PhD Studentship – Therapeutic genome editing – Paris area, France

PhD Studentship – Therapeutic genome editing –
Paris area, France

Universal genome editing strategies for treating Duchenne muscular dystrophy. INSERM U951 – Whitelab Genomics

Description: Duchenne muscular dystrophy (DMD; OMIM #310200) is a fatal X-linked neuromuscular disorder caused by loss of function mutations in the dystrophin gene affecting 1 in 5000 newborn males. DMD patients are restricted to wheelchair by the age of 12 years and usually succumb to cardiac or respiratory complications in their late twenties. Despite recent progress and extensive research over the last three decades, there is still no effective treatment for DMD. Gene Therapy (GT) trials using AAV to express functional truncated micro-dystrophin genes in muscles are currently on-going and therapeutic benefit still needs to be assessed. A promising alternative is based on upregulating surrogate genes able to compensate for the lack of dystrophin in DMD. We aim to combine CRISPR/Cas9 genome editing technology and genomic data mining to identify novel interesting therapeutic targets. Genetic animal model of DMD, available at the host laboratory, will be used to evaluate these new targets.

Host institute: The INSERM research unit U951-INTEGRARE ( Exploits gene-based technologies to investigate biological systems and pathologies of genetic origin and to design gene therapy treatments. The U951 partner with the university of Paris-Saclay and is hosted by Genethon (, non-profit biotherapy R&D organization dedicated to the development of GT products for rare human genetic diseases. WhiteLab Genomics ( develops state of the art software solutions using AI in order to accelerate and de-risk gene and cell therapy developments. Candidate’s

Qualifications: First and foremost, the desire to make an impact in the field of GT in a friendly, enthusiastic, dedicated and collaborative manner. Candidates should hold a master degree in biology/bioinformatics and should be able to work independently and interactively in a team setting, be responsible, organized and have a great work capacity and enthusiasm for research.

Closing Date: 30 September 2021

Value of award: Funding for the PhD fellowship is provided through the ‘’Paris Region PhD 2021’’ program.

Contact: Prospective candidates are strongly encouraged to email Dr. Mario Amendola

Please attach:
i) CV,
ii) full transcript of results (listing all subjects taken and their corresponding grades/marks),
iii) reference letter and
iv) a cover letter stating how the project meets your research interests.
Documents should be sent as a single pdf file